Development and validation of a chronic kidney disease progression model using patient-level simulations.

Chronic disease progression models are available for several highly prevalent conditions. For chronic kidney disease (CKD), the scope of existing progression models is limited to the risk of kidney failure and major cardiovascular (CV) events. The aim of this project was to develop a comprehensive CKD progression model (CKD-PM) that simulates the risk of CKD progression and a broad range of complications in patients with CKD.

The Impact of Unrelated Future Medical Costs on Economic Evaluation Outcomes for Different Models of Diabetes.

Objective: This study leveraged data from 11 independent international diabetes models to evaluate the impact of unrelated future medical costs on the outcomes of health economic evaluations in diabetes mellitus.

Methods: Eleven models simulated the progression of diabetes and occurrence of its complications in hypothetical cohorts of individuals with type 1 (T1D) or type 2 (T2D) diabetes over the remaining lifetime of the patients to evaluate the cost effectiveness of three hypothetical glucose improvement interventions versus a hypothetical control intervention. All models used the same set of costs associated with diabetes complications and interventions, using a United Kingdom healthcare system perspective.

Cost-effectiveness of empagliflozin as add-on to standard of care for chronic kidney disease management in the United Kingdom.

Objective: The sodium-glucose co-transporter-2 inhibitor empagliflozin was approved for treatment of adults with chronic kidney disease (CKD) on the basis of its demonstrated ability to slow CKD progression and reduce the risk of cardiovascular death. This analysis was performed to assess the cost-effectiveness of empagliflozin plus standard of care (SoC) vs SoC alone in the treatment of CKD in the UK.

Methods: A comprehensive, patient-level CKD progression model that simulates the evolution of risk factors for disease progression based on CKD-specific equations and clinical data was used to project a broad range of CKD-related complications.

Cost-effectiveness of the tubeless automated insulin delivery system vs standard of care in the management of type 1 diabetes in the United States.

A tubeless, on-body automated insulin delivery (AID) system (Omnipod 5 Automated Insulin Delivery System) demonstrated improved glycated hemoglobin A1c levels and increased time in range (70 mg/dL to 180 mg/dL) for both adults and children with type 1 diabetes in a 13-week multicenter, single-arm study. To assess the cost-effectiveness of the tubeless AID system compared with standard of care (SoC) in the management of type 1 diabetes (T1D) in the United States. Cost-effectiveness analyses were conducted from a US payer's perspective, using the IQVIA Core Diabetes Model (version 9.

The Cost-Effectiveness of Subcutaneous Semaglutide Versus Empagliflozin in Type 2 Diabetes Uncontrolled on Metformin Alone in Denmark.

Introduction: International and Danish guidelines recommend the use of glucagon-like peptide 1 receptor agonists (GLP-1 RA) and sodium-glucose cotransporter 2 (SGLT-2) inhibitors already in second line in the management of type 2 diabetes (T2D). The objective of this study was to evaluate the long-term cost-effectiveness (CE) of subcutaneous (SC) semaglutide (GLP-1 RA) versus empagliflozin (SGLT-2 inhibitor) in individuals with T2D uncontrolled on metformin alone from a Danish payer's perspective.

Methods: Cost-effectiveness analyses (CEA) were conducted from a Danish payer's perspective, using the IQVIA Core Diabetes model (CDM 9.

Exploring Structural Uncertainty and Impact of Health State Utility Values on Lifetime Outcomes in Diabetes Economic Simulation Models: Findings from the Ninth Mount Hood Diabetes Quality-of-Life Challenge.

Background: Structural uncertainty can affect model-based economic simulation estimates and study conclusions. Unfortunately, unlike parameter uncertainty, relatively little is known about its magnitude of impact on life-years (LYs) and quality-adjusted life-years (QALYs) in modeling of diabetes. We leveraged the Mount Hood Diabetes Challenge Network, a biennial conference attended by international diabetes modeling groups, to assess structural uncertainty in simulating QALYs in type 2 diabetes simulation models.

The cost-effectiveness of oral semaglutide versus empagliflozin in Type 2 diabetes in Denmark.

To evaluate the cost-effectiveness of oral semaglutide+metformin versus empagliflozin+metformin in people with Type 2 diabetes uncontrolled on msetformin alone. The IQVIA Core Diabetes Model was populated with efficacy data from a head-to-head study between oral semaglutide+metformin and empagliflozin+metformin. Danish costs and quality-of-life data were sourced from literature.

Cost-Effectiveness of iGlarLixi Versus iDegLira in Type 2 Diabetes Mellitus Inadequately Controlled by GLP-1 Receptor Agonists and Oral Antihyperglycemic Therapy.

Introduction: The fixed-ratio combinations (FRCs) of glucagon-like peptide 1 receptor agonists (GLP-1 RAs) and basal insulin, insulin glargine 100 U/mL plus lixisenatide (iGlarLixi), and insulin degludec plus liraglutide (iDegLira), have demonstrated safety and efficacy in patients with type 2 diabetes mellitus (T2DM) inadequately controlled on GLP-1 RAs. However, a comparative cost-effectiveness analysis between these FRCs from a UK Health Service perspective has not been conducted.

Methods: The IQVIA Core Diabetes Model was used to estimate lifetime costs and outcomes in patients with T2DM receiving iGlarLixi (based on the LixiLan-G trial) versus iDegLira (based on relative treatment effects from an indirect treatment comparison using data from DUAL III).

Cost-effectiveness of empagliflozin in patients with type 2 diabetes and established cardiovascular disease in China.

Background: In several cardiovascular outcome trials (CVOTs), empagliflozin (SGLT-2 inhibitor), sitagliptin (DPP-4 inhibitor) and liraglutide (GLP-1 receptor agonist) + standard of care (SoC) were compared to SoC in patients with type 2 diabetes and established cardiovascular disease (CVD). This study assessed the cost-effectiveness (CE) of empagliflozin + SoC in comparison to sitagliptin + SoC and liraglutide + SoC based on the respective CVOT.

Methods: The IQVIA Core Diabetes Model (CDM) was calibrated to reproduce the CVOT outcomes.

Potential clinical and economic impact of optimised maintenance therapy on discharged patients with COPD after hospitalisation for an exacerbation in China.

Objectives: Chronic obstructive pulmonary disease (COPD) exacerbations requiring hospitalisation are a considerable burden, both clinically and economically. Although long-acting maintenance therapy is recommended in both the GOLD (Global Initiative for Chronic Obstructive Lung Disease) and Chinese COPD guidelines, proper implementation is lacking. The objective of this study was to assess the clinical and economic impact of prescribing long-acting maintenance therapy to discharged patients with COPD after hospitalisation for an exacerbation in China by using an outcomes model.

Patient and caregiver productivity loss and indirect costs associated with cardiovascular events in Portugal.

Objectives: To present the Portuguese results of a multi-country cross-sectional survey aiming to estimate productivity loss in the first year after an acute coronary syndrome (ACS) or stroke.

Methods: Patients previously hospitalized for ACS or stroke were enrolled during a routine cardiology/neurology visit 3-12 months after the index event and ≥4 weeks after returning to work. Productivity loss for the patient and the caregiver in the previous four weeks were reported by the patient using the validated iMTA Productivity Cost Questionnaire (iPCQ).

Cost-effectiveness analysis of empagliflozin compared with glimepiride in patients with Type 2 diabetes in China.

The study assesses the cost-effectiveness of empagliflozin versus glimepiride in patients with Type 2 diabetes and uncontrolled by metformin alone in China, based on the EMPA-REG H2H-SU trial. A calibrated version of the IQVIA Core Diabetes Model was used. Cost of complications and utility were taken from literature.

Multicenter Prospective Evaluation of Parenteral Nutrition Preparation Time and Resource Utilization: 3-Chamber Bags Compared With Hospital Pharmacy-Compounded Bags.

Background: Parenteral nutrition (PN) is a complex and costly therapy that places significant demands on healthcare resources. Commercially manufactured 3-chamber bags (3CBs) offer potential time and cost advantages compared with hospital pharmacy-compounded bags (HCBs); however, no data are yet available from studies comparing these delivery systems in US hospitals. The primary aim of this study was to evaluate the PN preparation time and resource utilization required for 3CBs compared with HCBs in US hospitals.

Evaluating the Ability of Economic Models of Diabetes to Simulate New Cardiovascular Outcomes Trials: A Report on the Ninth Mount Hood Diabetes Challenge.

Objectives: The cardiovascular outcomes challenge examined the predictive accuracy of 10 diabetes models in estimating hard outcomes in 2 recent cardiovascular outcomes trials (CVOTs) and whether recalibration can be used to improve replication.

Methods: Participating groups were asked to reproduce the results of the Empagliflozin Cardiovascular Outcome Event Trial in Type 2 Diabetes Mellitus Patients (EMPA-REG OUTCOME) and the Canagliflozin Cardiovascular Assessment Study (CANVAS) Program. Calibration was performed and additional analyses assessed model ability to replicate absolute event rates, hazard ratios (HRs), and the generalizability of calibration across CVOTs within a drug class.

Long-Term Cost-Effectiveness Analyses of Empagliflozin Versus Oral Semaglutide, in Addition to Metformin, for the Treatment of Type 2 Diabetes in the UK.

Introduction: International guidelines recommend treatment with a sodium-glucose cotransporter-2 (SGLT-2) inhibitor or glucagon-like peptide-1 (GLP-1) receptor agonist for treatment intensification in type 2 diabetes mellitus (T2DM) patients with progression on metformin. In the randomised, controlled, Peptide Innovation for Early Diabetes Treatment (PIONEER) 2 trial, the SGLT-2 inhibitor empagliflozin was compared with the GLP-1 receptor agonist oral semaglutide, in addition to metformin. The aim of the current study was to assess the long-term cost-effectiveness of empagliflozin 25 mg versus oral semaglutide 14 mg, in addition to metformin, for T2DM patients in the UK.

Cost-effectiveness of empagliflozin compared with liraglutide based on cardiovascular outcome trials in Type II diabetes.

Cost-effectiveness (CE) analysis of empagliflozin+standard of care (SoC) compared with SoC and liraglutide+SoC, in patients with Type II diabetes and established cardiovascular disease, was conducted using evidence from cardiovascular outcomes trials. The IQVIA Core Diabetes Model was calibrated to predict same outcomes observed in EMPA-REG OUTCOME and LEADER trials. Three-year observed cardiovascular events of SoC, empagliflozin+SoC and liraglutide+SoC were derived from EMPA-REG OUTCOME trial and an indirect comparison.

Cost-Effectiveness Analysis of Empagliflozin in Comparison to Sitagliptin and Saxagliptin Based on Cardiovascular Outcome Trials in Patients with Type 2 Diabetes and Established Cardiovascular Disease.

Introduction: In the cardiovascular outcome trials (CVOT) EMPA-REG OUTCOME, TECOS and SAVOR-TIMI 53, empagliflozin [sodium/glucose cotransporter 2 (SGLT2) inhibitor], sitagliptin and saxagliptin [both dipeptidyl peptidase 4 (DPP4) inhibitors] + standard of care (SoC) were compared to SoC in patients with type 2 diabetes and established cardiovascular disease (CVD). This study assessed the cost-effectiveness (CE) of empagliflozin + SoC in comparison to sitagliptin + SoC and saxagliptin + SoC based on the respective CVOT.

Methods: The IQVIA Core Diabetes Model (CDM) was calibrated to reproduce the CVOT outcomes.

Cost-effectiveness of physical activity in the management of COPD patients in the UK.

Background: While the Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines advise exercise to reduce disease progression, little investment in promoting physical activity (PA) is made by health care authorities. The purpose of this study was to estimate the cost-effectiveness of regular PA vs sedentary lifestyle in people with COPD in the UK.

Methods: Efficacy, quality of life, and economic evidence on the PA effects in COPD patients were retrieved from literature to serve as input for a Markov microsimulation model comparing a COPD population performing PA vs a COPD population with sedentary lifestyle.

Cost-effectiveness analysis of the SAPIEN 3 TAVI valve compared with surgery in intermediate-risk patients.

Objective: Transcatheter aortic valve implantation (TAVI) has become the therapy of choice for treating severe aortic stenosis in patients at high-risk for surgery or where it is considered too risky to attempt. This uptake varies across geographies however, and its cost or value has frequently been cited as the reason for this. We sought to evaluate the potential cost and clinical impact of TAVI in intermediate risk patients from a French collective perspective.

Assessing the Burden of Type 2 Diabetes in China Considering the Current Status-Quo Management and Implications of Improved Management Using a Modeling Approach.

Background: Recent estimates from the International Diabetes Federation Diabetes Atlas have quantified the total annual expenditure for diabetes in China to be between 354 and 611 billion Chinese yuan (¥) (2015).

Objectives: To use a modeling approach to assess the current and possible future diabetes burden in China on the basis of the current standard of type 2 diabetes (T2D) management (status quo [SQ]) and a series of hypothetical improved management strategies.

Methods: The IQVIA CORE Diabetes Model was used to evaluate the economic burden of T2D in China on the basis of assumptions reflecting the current SQ of T2D management and a number of stepwise improvements.

Cost-Effectiveness of rFVIIa versus pd-aPCC in the Management of Mild to Moderate Bleeds in Pediatric Patients with Hemophilia A with Inhibitors in Mexico.

Objective: To compare the costs and clinical consequences of treating mild-to-moderate joint bleeds with recombinant activated factor VII (rFVIIa) versus plasma-derived activated prothrombin complex concentrate (pd-aPCC) in pediatric patients with hemophilia A with inhibitors in Mexico.

Methods: A cost-effectiveness model was developed using TreeAge Pro v14.2.

Cost versus utility of aclidinium bromide 400 µg plus formoterol fumarate dihydrate 12 µg compared to aclidinium bromide 400 µg alone in the management of moderate-to-severe COPD.

Purpose: Aclidinium-formoterol 400/12 µg is a long-acting muscarinic antagonist (LAMA) and a long-acting β2-agonist in a fixed-dose combination used in the management of patients with COPD. This study aimed to assess the cost-effectiveness of aclidinium-formoterol 400/12 µg against the long-acting muscarinic antagonist aclidinium bromide 400 µg.

Materials And Methods: A five-health-state Markov transition model with monthly cycles was developed using MS Excel to simulate patients with moderate-to-severe COPD and their initial lung-function improvement following treatment with aclidinium-formoterol 400/12 µg or aclidinium 400 µg.

Distinct higher-order α-synuclein oligomers induce intracellular aggregation.

Misfolding and aggregation of α-synuclein (α-syn) into Lewy bodies is associated with a range of neurological disorders, including Parkinson's disease (PD). The cell-to-cell transmission of α-syn pathology has been linked to soluble amyloid oligomer populations that precede Lewy body formation. Oligomers produced in vitro under certain conditions have been demonstrated to induce intracellular aggregation in cell culture models.